Safety and Efficacy Study of EXC 001 to Improve the Appearance of Scars in Subjects Undergoing Elective Abdominoplasty

A PHASE 2, RANDOMIZED, DOUBLE-BLIND, WITHIN-SUBJECT CONTROLLED STUDY TO EVALUATE EFFICACY AND SAFETY OF EXC 001 FOR THE AMELIORATION OF SCARRING OF THE SURGICAL INCISION IN SUBJECTS UNDERGOING AN ELECTIVE ABDOMINOPLASTY

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01037985

Enrollment

Registered

2009-12-23

Start date

2009-12-03

Completion date

2010-09-22

Last updated

2021-08-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Scar Prevention

Keywords

Scarring, Cicatrix, Fibrosis, Pathologic Process

Brief summary

This study will compare how well EXC 001 works versus placebo in reducing the appearance of scars in subjects undergoing elective abdominoplasty. The study will also evaluate the safety of EXC 001 in healthy adult subjects.

Interventions

DRUGEXC 001

Multiple intradermal injections of EXC 001 and placebo

DRUGPlacebo

Multiple intradermal injections of EXC 001 and placebo

Study design

Allocation

RANDOMIZED

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

ALL

Age

18 Years to 45 Years

Healthy volunteers

Yes

Inclusion criteria

* Subjects must have sufficient excess abdominal tissue to qualify for a standard elective abdominoplasty * Subject has chosen to have an elective abdominoplasty * Medically healthy with normal screening results * Subjects must not be pregnant or lactating

Exclusion criteria

* Females who are currently pregnant or pregnant during the 12 months prior to inclusion in the study, or lactating * Participation in another clinical trial within 30 days prior to the start of the study * Any other condition or prior therapy, which, in the opinion of the PI, would make the subject unsuitable for this study

Design outcomes

Primary

Measure	Time frame	Description
Expert Panel Scar Assessment Score at Week 12: Part B	Week 12	Scar assessment by an expert panel was done on blinded photographs using 100 millimeter (mm) visual analog scale (VAS) where a score of 0 = best possible scar and a score of 100 = worst possible scar. A pair of photographs for each participant were presented and evaluated 3 times by an expert panel.

Secondary

Measure	Time frame	Description
Physician Observer Scar Assessment Score at Week 12 and 24: Part B	Week 12 and 24	Physician assessment of scar was done using a valid published 10-point rating scale. Physician rated vascularity, pigmentation, thickness, relief, pliability, surface area and overall opinion for a scar on a score of 1 = normal skin to 10 = worst scar imaginable. Composite score was the sum of all the scores except the overall opinion score and range from 6 (best score) to 60 (worst score).
Participants Observer Scar Assessment Score at Week 12 and 24: Part B	Week 12 and 24	Participants rated pain, itching, color, stiffness, thickness, irregularity, and overall opinion of scar on 10-point scale. For pain and itching associated with scar: range = 1 (no, not at all) to 10 (yes, worst imaginable) and for other parameters associated with scar compared to normal skin: range = 1 (no, same as normal skin) to 10 (yes, very different). Composite score = sum of all scores except overall opinion, range 6 (best) to 60 (worst). Scar appearance composite score = sum of all scores except overall opinion, pain and itching, range 4 (best) to 40 (worst).
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	Day 1 of Part A up to Week 24 of Part B	An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included SAEs and all non-SAEs that occurred during the study.
Number of Participants With Abnormal Physical Examinations Findings: Part A and Part B	Day 1 of Part A up to Week 12 of Part B	Physical examination included the assessment of skin; head, ears, eyes, nose, and throat; respiratory; cardiovascular; abdomen; musculoskeletal; neurological; gastrointestinal; genitourinary; endocrine and lymph nodes. Abnormal physical examinations findings was based on investigator's discretion.
Expert Panel Scar Assessment Score at Week 24: Part B	Week 24	Scar assessment by an expert panel was done on blinded photographs using 100 mm VAS where a score of 0 = best possible scar and a score of 100 = worst possible scar. A pair of photographs for each participant was presented and evaluated 3 times by an expert panel.
Number of Participants With Clinically Significant Findings in Laboratory Examinations: Part A and Part B	Day 1 of Part A up to Week 12 of Part B	Laboratory analysis included hematology, biochemistry and urinalysis. Hematology range: basophils (bas) 0-0.2, eosinophils (eos) 0-0.4, leukocytes (leu) 4-10.5, lymphocytes (lym) 0.7-4.5, neutrophils (neu) 1.8-7.8, platelet 140-415, monocytes (mon) 0.1-1 in 10\^9 per liter; bas/leu 0-3, eos/leu 0-7, lym/leu 14-46, mon/leu 4-13, neu/leu and neu/leu 40-74 in percentage, erythrocytes 3.8-5.1 10\^12/L, hematocrit 0.34-0.44 L/L, hemoglobin 115-150 gram per liter (g/L). Biochemistry range: creatine kinase 24-173, alkaline phosphatase 25-150, alanine aminotransferase (AT) and aspartate AT 0-40 in International units per liter; creatinine 50-88, urate 89-399, bilirubin 2-21 in micromole per liter, glucose 3.6-5.5, potassium 3.5-5.5, sodium 135-148, blood urea nitrogen 1.8-9.3 in millimole/L, albumin 35-55 g/L. Urinalysis parameters: pH (5-7.5), specific gravity (1.005-1.03). Participants with clinically significant findings were reported.
Number of Participants With Clinically Significant Findings in Vital Signs and Weight: Part A and Part B	Day 1 of Part A up to Week 12 of Part B	Following vital sign parameters were assessed: diastolic blood pressure, systolic blood pressure, respiration rate, pulse rate, and temperature. Number of participants with clinically significant change in any vital sign parameter and weight compared to baseline were reported. Clinical significance was based on investigator's discretion.
Number of Participants With Positive Skin Sensitivity Reaction: Part A and Part B	Day 21 of Part A up to Day 14 of Part B	Participants were instructed to inform the investigator in case of any itching, redness, pain or any other symptom that appears to be a rash at the injection sites. Erythematous, raised (indurated) and edematous reactions were considered as positive skin sensitivity reactions.
Number of Participants With Clinically Significant Findings in Electrocardiogram (ECG): Part A and Part B	Day 1 of Part A up to Week 12 of Part B	Number of participants with clinically significant abnormality in ECG were reported. Clinical significance was based on investigator's discretion.

Countries

United States

Participant flow

Recruitment details

Participants who had chosen and qualified (sufficient excess abdominal tissue) for a standard elective abdominoplasty were recruited in this study. Study had 2 parts- Part A and B. Participants with negative skin sensitization in Part A (skin testing) were assigned to Part B (abdominoplasty surgery) of the study.

Participants by arm

Arm	Count
All Enrolled Participants All participants who were enrolled in the study.	41
Total	41

Withdrawals & dropouts

Period	Reason	FG000	FG001
Part A: Skin Testing (50 Days)	Physician Decision	1	0
Part A: Skin Testing (50 Days)	Withdrawal by Subject	3	0
Part B:Abdominoplasty Surgery (24 Weeks)	Lost to Follow-up	0	1
Part B:Abdominoplasty Surgery (24 Weeks)	Positive Skin Sensitization	0	4

Baseline characteristics

Characteristic	All Enrolled Participants
Age, Continuous	34.7 years STANDARD_DEVIATION 4.68
Sex: Female, Male Female	41 Participants
Sex: Female, Male Male	0 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk
deaths Total, all-cause mortality	— / —	— / —	— / —
other Total, other adverse events	7 / 41	18 / 33	3 / 32
serious Total, serious adverse events	0 / 41	0 / 33	0 / 32

Outcome results

Primary

Expert Panel Scar Assessment Score at Week 12: Part B

Scar assessment by an expert panel was done on blinded photographs using 100 millimeter (mm) visual analog scale (VAS) where a score of 0 = best possible scar and a score of 100 = worst possible scar. A pair of photographs for each participant were presented and evaluated 3 times by an expert panel.

Time frame: Week 12

Population: Completer population included all participants who had missed not more than 1 dose of study drug, had the Week 12 assessment and non-missing data for VAS scar assessment score.

Arm	Measure	Value (MEAN)	Dispersion
EXC 001 (PF-0647387) During Part B	Expert Panel Scar Assessment Score at Week 12: Part B	37.8 millimeter	Standard Deviation 12.96
Placebo During Part B	Expert Panel Scar Assessment Score at Week 12: Part B	42.1 millimeter	Standard Deviation 12.52

Comparison: Within participant treatment difference was assessed between the treatment regimens each participant received (EXC 001 minus placebo).p-value: 0.16895% CI: [-10.4, 1.9]t-test, 2 sided

Secondary

Expert Panel Scar Assessment Score at Week 24: Part B

Scar assessment by an expert panel was done on blinded photographs using 100 mm VAS where a score of 0 = best possible scar and a score of 100 = worst possible scar. A pair of photographs for each participant was presented and evaluated 3 times by an expert panel.

Time frame: Week 24

Population: Completer population included all participants who had missed not more than 1 dose of study drug, had the Week 12 assessment and non-missing data for VAS scar assessment score. Here 'N' (number of participants analyzed) signifies those participants who were evaluable for this outcome measure.

Arm	Measure	Value (MEAN)	Dispersion
EXC 001 (PF-0647387) During Part B	Expert Panel Scar Assessment Score at Week 24: Part B	37.9 millimeter	Standard Deviation 9.29
Placebo During Part B	Expert Panel Scar Assessment Score at Week 24: Part B	37.6 millimeter	Standard Deviation 10.73

Comparison: Within participant treatment difference was assessed between the treatment regimens each participant received (EXC 001 minus placebo).p-value: 0.85895% CI: [-3.5, 4.2]t-test, 2 sided

Secondary

Number of Participants With Abnormal Physical Examinations Findings: Part A and Part B

Physical examination included the assessment of skin; head, ears, eyes, nose, and throat; respiratory; cardiovascular; abdomen; musculoskeletal; neurological; gastrointestinal; genitourinary; endocrine and lymph nodes. Abnormal physical examinations findings was based on investigator's discretion.

Time frame: Day 1 of Part A up to Week 12 of Part B